NL-OMON53772已完成3 期
A Phase 3, Randomized, Double-Blind Trial of Two Formulations of Setmelanotide (Daily and Weekly) with a Crossover to Open-Label Once Weekly Setmelanotide in Patients with Specific Gene Defects in the Melanocortin-4 Receptor Pathway Who Are Currently on a Stable Dose of the Once Daily Formulation - Setmelanotide - Daily vs weekly with crossover to open label
适应症
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 3
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- 2 至 64（—）
入选标准
- •1. All patients must have met the criteria for diagnosis of a gene defect in
- •the MC4R pathway (BBS, biallelic PPL, heterozygous PPL), for which they are
- •being treated with QD setmelanotide.
- •2. Patients must be >=6 years old at screening.
- •3. Patients must have been taking the setmelanotide QD formulation for at least
- •6 months in the LTE trial with acceptable safety and tolerability, and the dose
- •level must have been stable at 2, 2.5 or 3 mg of setmelanotide for at least the
- •last 3 months prior to starting the Run-in Period.
- •4. Patient and/or parent or guardian is able to communicate well with the
- •Investigator, to understand and comply with the requirements of the trial and
- •is able to understand and sign the written informed consent/assent.
- •5. Patient must meet one of the following requirements:
- •Female participants of childbearing potential, defined as fertile, following
- •menarche and until becoming post-menopausal unless permanently sterile
- •(hysterectomy, bilateral salpingectomy, or bilateral oophorectomy), must be
- •confirmed non-pregnant and agree to use a highly effective form of
- •contraception throughout the trial and for 90 days following the trial. Highly
- •effective forms of contraception are detailed below and in Section 8.8.7:
- •- Combined (estrogen and progestogen containing) hormonal contraception
- •associated with inhibition of ovulation (oral, intravaginal, or transdermal)
- •- Progestin-only hormonal contraception associated with inhibition of ovulation
- •(oral, implantable, or injectable)
- •- Intrauterine device (IUD)
- •- Intrauterine hormone-releasing system
- •- Bilateral tubal occlusion
- •- Vasectomy/vasectomized partner (provided that the vasectomized partner is the
- •sole sexual partner of the female participant, and the vasectomized partner has
- •received medical assessment of surgical success)
- •- Sexual abstinence, only if it is the preferred and usual lifestyle of the
- •Female participants of non-childbearing potential, defined as: permanently
- •sterile (status post hysterectomy, bilateral oophorectomy, or bilateral
- •salpingectomy), or post-menopausal for at least 12 months (and confirmed with a
- •screening follicle-stimulating hormone (FSH) level in the post-menopausal lab
- •range) do not require contraception during the trial.
- •Younger female patients who have not achieved sexual maturity at study entry
- •will be assessed for Tanner staging and required to comply with contraception
- •requirements at first menarche.
- •Male participants with female partners of childbearing potential must agree to
- •use a highly effective method of contraception if they become sexually active
- •during the trial or within 90 days following their participation in the study.
- •Male patients must also not donate sperm during and for 90 days following their
- •participation in the trial.
排除标准
- •1. HbA1C >9.0% at screening.
- •2. Has taken a medication that is approved to treat obesity (e.g., orlistat,
- •lorcaserin, phentermine-topiramate, naltrexone-bupropion) within 3 months prior
- •to starting the Run-in Period. Glucagon-like peptide-1 (GLP) -1) receptor
- •agonists being prescribed for the treatment of obesity are not allowed.
- •3. History of significant liver disease or liver injury, or a current liver
- •assessment due to abnormal liver tests for an etiology other than nonalcoholic
- •fatty liver disease (NAFLD). Thus, any underlying etiology besides NAFLD,
- •including diagnosed nonalcoholic steatohepatitis (NASH), other causes of
- •hepatitis, or history of hepatic cirrhosis is exclusionary, but the presence of
- •NAFLD is not exclusionary.
- •4. Moderate to severe renal dysfunction as defined by a glomerular filtration
- •rate <30 mL/min. (based upon the Chronic Kidney Disease Epidemiology
- •Collaboration [CKD-EPI] creatinine equation 2021 from the National Kidney
- •Foundation). In patients <18 years of age the Bedside Schwartz Equation should
- •be used to calculate estimated glomerular filtration rate (eGFR).
- •5. Significant dermatologic findings relating to melanoma or pre-melanoma skin
- •lesions (excluding non-invasive basal or squamous cell lesion), determined as
- •part of comprehensive skin evaluation performed by the Investigator during
- •screening. Any concerning lesions identified during the Screening Period will
- •be biopsied and results must be known to be benign prior to enrollment. If the
- •pretreatment biopsy results are of concern, the patient should be excluded from
- •6. Diagnosis of schizophrenia, bipolar disorder, personality disorder, or other
- •psychiatric disorders that the Investigator believes will interfere
- •significantly with trial compliance. Neurocognitive disorders affecting ability
- •to consent will not be disqualifying as long as an appropriate guardian able to
- •give consent has been appointed.
- •7. Clinically significant depression or suicidality as defined by: any suicidal
- •ideation of type 4 or 5 on the C SSRS, any lifetime history of a suicide
- •attempt, or any suicidal behavior in the last month or a Patient Health
- •Questionnaire-9 (PHQ-9) score of >=15 during Screening in patients with no
- •significant neurocognitive deficits.
- •8. Patient is not suitable, in the opinion of the Investigator, to participate
- •in the trial.
- •9. Hypersensitivity to the active substance or to any of the excipients of the
- •investigational products (active or placebo).
- •10. Inability to comply with the QW and QD injection regimens.
- •11. Participation in any clinical trial with an investigational drug/device
- •within 3 months prior to the first day of dosing, with the exception of a
- •setmelanotide clinical trial.
- •12. Legally protected persons per local regulations (e.g., those that fall
- •under the L1121-6 article of the Public Health code in France) or other
- •applicable local laws.
- •13. The patient or a relative of the patient is the investigator or a sub
- •investigator, research assistant, pharmacist, trial coordinator, or other
- •staff directly involved with the conduct of the trial.
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